Protein Homeostasis Diseases: Mechanisms and Novel Therapies 1st Edition by Angel Pey ISBN 0128191333 9780128191330

Section I: Introduction of Protein Folding and Homeostasis

• Chapter 1: Protein Folding – How, Why, and Beyond

  • Abstract
  • Abbreviations
  • Introduction
  • Acknowledgments

• Chapter 2: Protein Homeostasis and Disease

  • Abstract
  • Abbreviations
  • Protein Folding In Vitro and In Vivo
  • Human Misfolding Diseases
  • Acknowledgments
  • Conflict of Interest
  • Funding

Section II: Protein Folding and Homeostasis at the Organismal and Proteomic Scales

• Chapter 3: Caenorhabditis elegans as a Model for Protein Homeostasis Diseases

  • Abstract
  • Abbreviations
  • C. elegans as a Model Organism
  • Conclusion

• Chapter 4: Proteome-Scale Studies of Protein Stability

  • Abstract
  • Abbreviations
  • Introduction
  • Protein Stability and Unfolding
  • Methods to Measure Protein Stability In Vitro and In Vivo
  • Biological Readouts for Stability
  • Structural and Proteome-Scale Analyses
  • Applications to Biomedical Research
  • Conclusions
  • Acknowledgments

• Chapter 5: Classifying Disease-Associated Variants by Protein Activity and Stability

  • Abstract
  • Abbreviations
  • Introduction
  • PTEN Variant Selection
  • Comparing Assays and Predictions for Variant Effects
  • Stability Loss and PTEN Function
  • Conclusions
  • Methods
  • Acknowledgments

Section III: Disturbance in Protein Homeostasis in Disease – Genetics, Mechanisms, and Modulation by Natural Ligands

• Chapter 6: Protein Destabilization and Degradation in Hereditary Disease

  • Abstract
  • Abbreviations
  • Protein Quality Control in Hereditary Diseases
  • Folding, Refolding, and Degradation Pathways
  • Therapeutic Approaches
  • Acknowledgments
  • Conflict of Interest
  • Funding

• Chapter 7: Detection of Amyloid Aggregation in Living Systems

  • Abstract
  • Abbreviations
  • Techniques for In Vivo Detection
  • Animal Models for Testing
  • Insights into Neurodegeneration
  • Future Outlook
  • Acknowledgments

• Chapter 8: Molecular Mechanisms of Amyloid Aggregation in Human Proteinopathies

  • Abstract
  • Abbreviations
  • Aggregates and Amyloid Fibrils
  • Mechanisms and Disease Implications
  • Concluding Remarks
  • Acknowledgments

• Chapter 9: Metals and Toxic Mechanisms in Neurodegenerative Diseases

  • Abstract
  • Abbreviations
  • Metal Ions in Neurodegeneration
  • Zinc and Alzheimer’s Disease Aggregates
  • Metal Chelation Therapies
  • Acknowledgments

• Chapter 10: Vitamin B6-Dependent Enzymes and Disease

  • Abstract
  • Abbreviations
  • Misfolding Diseases and Natural Ligands
  • Vitamin B6 and Associated Disorders
  • Hyperoxaluria and AGT Deficiency
  • Conclusions
  • Acknowledgments

• Chapter 11: Galactosemia – Opportunities for Novel Therapies

  • Abstract
  • Abbreviations
  • Types of Galactosemia
  • Pathology, Treatment, and Novel Approaches
  • Pharmacological Chaperones for GALT Deficiency
  • Conclusions
  • Acknowledgments

• Chapter 12: Protein Homeostasis in GPCR Intracellular Trafficking

  • Abstract
  • Abbreviations
  • Proteostasis in GPCR Trafficking
  • Targeting Misfolded GPCRs with Chaperones
  • Conclusions
  • Acknowledgments

Section IV: Novel Small Molecules for Correcting Human Protein Diseases

• Chapter 13: Structure-Guided Discovery of Pharmacological Chaperones

  • Abstract
  • Abbreviations
  • Understanding Disease Variants Structurally
  • Primary Screening for Chaperones
  • Concluding Remarks
  • Statements
  • Acknowledgments

• Chapter 14: Virtual Screening in Drug Discovery

  • Abstract
  • Abbreviations
  • Drug Discovery Process
  • Binding Affinity and Scoring Functions
  • Screening Protocols for Receptor–Ligand Binding

• Chapter 15: Differential Scanning Fluorimetry in Pharmacological Screening

  • Abstract
  • Abbreviations
  • Background and High-Throughput Screening
  • Conclusion
  • Acknowledgments

• Chapter 16: Cellular High-Throughput Screening

  • Abstract
  • Abbreviations
  • Introduction to Screening Techniques
  • Genetic Diseases and High-Throughput Screening
  • Conclusion
  • Acknowledgments

• Chapter 17: High-Throughput Screening for Intrinsically Disordered Proteins

  • Abstract
  • Abbreviations
  • Techniques: Fluorescence, NMR, SPR
  • Applications for Identifying Potential Drug Hits
  • Funding

• Chapter 18: Chaperones in Protein Degradation and Erythropoietic Porphyria

  • Abstract
  • Abbreviations
  • UROIIIS Stability Defects
  • Congenital Erythropoietic Porphyria